Deaf baby girl hears for first time after 'spectacular' gene therapy at Addenbrooke's Hospital

WATCH REBECCA HOWARTH'S REPORT ON OPAL AND HER FAMILY

A baby girl born completely deaf can hear for the first time after receiving ground-breaking gene therapy.

Opal Sandy from Oxfordshire was eleven months old when she was treated at Addenbrooke’s Hospital in Cambridge.

She is the first British patient and the youngest child to receive this type of treatment.

Opal was born deaf because of a rare genetic condition, auditory neuropathy, caused by the disruption of nerve impulses travelling from the inner ear to the brain.

Within four weeks of having the gene therapy infusion to her right ear, Opal responded to sound, even with the cochlear implant in her left ear switched off.

Clinicians noticed continuous improvement in Opal’s hearing in the weeks afterwards.

At 24 weeks, they confirmed Opal had close to normal hearing levels for soft sounds, such as whispering, in her treated ear.

Now 18 months old, Opal can respond to her parents’ voices and can communicate words such as “Dada” and “bye-bye.”

Opal’s mother, Jo Sandy, said: “When Opal could first hear us clapping unaided it was mind-blowing.

"We were so happy when the clinical team confirmed at 24 weeks that her hearing was also picking up softer sounds and speech.

"The phrase ‘near normal’ hearing was used and everyone was so excited such amazing results had been achieved.”

Opal was being treated in a global gene therapy trial known as CHORD.

Professor Manohar Bance, an ear surgeon at Cambridge University Hospitals NHS Foundation Trust and chief investigator of the trial, described the results as "spectacular".

"Gene therapy has been the future of otology and audiology for many years and I’m so excited that it is now finally here." he said.

"This is hopefully the start of a new era for gene therapies for the inner ear and many types of hearing loss.”

Opal was given an infusion containing a harmless virus, delivered via an injection in the cochlea during surgery under general anaesthesia.

During surgery, while Opal was given the gene therapy in right ear, a cochlear implant was fitted in her left ear.

James Sandy, Opal’s father, said: “It was our ultimate goal for Opal to hear all the speech sounds.

"It’s already making a difference to our day-to-day lives, like at bath-time or swimming, when Opal can’t wear her cochlear implant.

"We feel so proud to have contributed to such pivotal findings, which will hopefully help other children like Opal and their families in the future.”

Opal’s 24-week results, alongside other scientific data from the CHORD trial are being presented at the American Society of Gene and Cell Therapy (ASGC) in Baltimore, USA this week.

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